MUSCULAR DYSTROPHIES

This program provides access to an experimental drug called AOC 1044 for people with Duchenne muscular dystrophy who have specific genetic mutations. The treatment aims to help muscles produce a more functional protein by 'skipping' a problematic section of genetic code. Eligible…

This study is testing a brain implant system to help people who have lost the ability to speak due to conditions like ALS, spinal cord injury, or stroke. The implant reads brain signals related to speech, aiming to let participants control a computer to communicate with their tho…

This study is testing a tiny brain implant that could help people who have lost the ability to speak due to conditions like ALS, stroke, or spinal cord injury. The device reads brain signals when a person tries to talk and translates them into computer-generated speech. Researche…

This study is testing a new oral medicine called SAT-3247 for boys aged 7 to under 10 who have Duchenne Muscular Dystrophy (DMD) and can still walk. The main goals are to find a safe and well-tolerated dose and to see if the medicine can help improve muscle strength and function.…

This study aims to better understand how fatigue affects walking in people with neuromuscular diseases like muscular dystrophy and spinal muscular atrophy. Researchers will observe 120 adults who can walk independently during a clinic walking test and for one week at home using a…

This study aims to better understand inherited nerve and muscle disorders that begin in childhood, like certain muscular dystrophies. Researchers will follow 9,300 participants—including patients, their family members, and healthy volunteers—over years to track how symptoms chang…